Cargando…
Gaucher Disease in Bone: From Pathophysiology to Practice
Gaucher disease (GD) is a rare, genetic lysosomal disorder leading to lipid accumulation and dysfunction in multiple organs. Involvement of the skeleton is one of the most prevalent aspects of GD and a major cause of pain, disability, and reduced quality of life. Uniform recommendations for contempo...
Autores principales: | Hughes, Derralynn, Mikosch, Peter, Belmatoug, Nadia, Carubbi, Francesca, Cox, TimothyM, Goker‐Alpan, Ozlem, Kindmark, Andreas, Mistry, PramodK, Poll, Ludger, Weinreb, Neal, Deegan, Patrick |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2019
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6852006/ https://www.ncbi.nlm.nih.gov/pubmed/31233632 http://dx.doi.org/10.1002/jbmr.3734 |
Ejemplares similares
-
Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials
por: Elstein, Deborah, et al.
Publicado: (2022) -
Demographics and patient characteristics of 1209 patients with Gaucher disease: Descriptive analysis from the Gaucher Outcome Survey (GOS)
por: Zimran, Ari, et al.
Publicado: (2017) -
Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS)
por: Hughes, Derralynn A., et al.
Publicado: (2022) -
Optimal therapy in Gaucher disease
por: Goker-Alpan, Ozlem
Publicado: (2010) -
Reply to Mistry et al. The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on “Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158”
por: Hughes, Derralynn A., et al.
Publicado: (2023)